Actively Recruiting
Lentiviral Gene Therapy for CGD
Led by Shenzhen Geno-Immune Medical Institute · Updated on 2026-04-24
10
Participants Needed
1
Research Sites
234 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a Phase I/II clinical trial of gene therapy for treating Chronic Granulomatous Disease using a high-safety, high-efficiency, self-inactivating lentiviral vector TYF to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the TYF-CGD gene transfer clinical protocol.
CONDITIONS
Official Title
Lentiviral Gene Therapy for CGD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with Chronic Granulomatous Disease (CGD) of any age
- Molecular diagnosis confirmed by DNA sequencing supported by lab evidence showing absent or significantly reduced NADPH-oxidase activity
- Karnofsky-Index score of 70% or higher
- At least one prior, ongoing, or refractory severe infection or inflammatory complication requiring hospitalization despite drug treatment
- Written informed consent from adult patients, and assent for pediatric subjects aged seven years or older
You will not qualify if you...
- Contraindications for leukapheresis such as anemia with hemoglobin less than 8 g/dl, cardiovascular instability, or severe coagulopathy
- Contraindications for administration of conditioning medication
- Female patients who are pregnant or breastfeeding as confirmed by history or positive pregnancy test
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China, China
Actively Recruiting
Research Team
L
Lung-Ji Chang, Ph.D
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here